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Hepatic Veno-Occlusive DiseasePhase 3Treatment INDNCT00628498

Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

A Phase 3 study testing Defibrotide in people with Hepatic Veno-Occlusive Disease, run by Jazz Pharmaceuticals at 125 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). Read more about this study

Who is it for?
Any age
Conditions the study is for
Hepatic Veno-Occlusive Disease
What is being tested?
Defibrotide
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1,206 participants
Where
125 sitesUnited States
Study dates
Dec 2007to Sep 2016
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Entry criteria include the following:
  • Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:
  • 1.1 Baltimore Criteria- Bilirubin ≥2 mg/dL and at least 2 of the following clinical findings:
  • Ascites (radiographic or physical exam)
  • Weight gain of ≥5% compared to the day of conditioning-- if this value is not available, the weight on the date of admission to the SCT unit may be used)
  • Hepatomegaly; increased over baseline.
Show all 12Show fewer
  • 1.2 Modified Seattle Criteria: At least two of the following
  • Bilirubin ≥2 mg/dL
  • Ascites (radiographic or physical exam) and/or weight gain ≥5% above baseline weight (defined as weight on the first day of conditioning- if this value is not available, the weight on the date of admission to the SCT unit may be used)
  • hepatomegaly increased over baseline
  • 1.3 Patients that do not meet the Baltimore Criteria or Modified Seattle Criteria and have biopsy proven VOD are eligible.
  • Patient must also provide written informed consent.

Who cannot join

  • Use of any medication which increases the risk of hemorrhage is disallowed. Use of heparin or other anticoagulants is disallowed within 12 hours unless being used for routine central venous line management, fibrinolytic instillation for central venous line occlusion, intermittent dialysis or ultrafiltration of CVVH.
  • Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells (e.g., a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss, OR bleeding from a site which in the Investigator's opinion constitutes a potential life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of amount of blood loss, at any point from the date of SCT through the date of severe VOD diagnosis.
  • Hemodynamic instability as defined by a requirement for multiple pressors, or inability to maintain mean arterial pressure (for children: to maintain mean arterial pressure within 1 standard deviation of age-adjusted levels) with single pressor support.
  • Woman who are pregnant.

Trial locations and contacts

125 sites in 1 country.

125 sites in 1 country

United States125 sites

  • Children's Hospital Medical

    Akron, Ohio, 44308

  • Presbyterian Hospital

    Albuquerque, New Mexico, 87106

  • University of Michigan Health System

    Ann Arbor, Michigan, 48109

  • Childrens Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Emory University Hospital

    Atlanta, Georgia, 30322

  • Northside Hospital

    Atlanta, Georgia, 30342

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • University of Maryland - Greenebaum Cancer Center

    Baltimore, Maryland, 21201

  • Johns Hopkins University

    Baltimore, Maryland, 21287

  • National Institutes of Health

    Bethesda, Maryland, 20892

  • Children's Hospital of Alabama

    Birmingham, Alabama, 35233

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Boston Children's Hospital DFCI/CHB-Pediatrics

    Boston, Massachusetts, 02115

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

  • The University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Levine Children's Hospital

    Charlotte, North Carolina, 28203

  • University of Virginia Health System

    Charlottesville, Virginia, 22908

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Northwest Memorial Hospital

    Chicago, Illinois, 60611

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • Pediatric Hematology/Oncology MC4060 - University of Chicago

    Chicago, Illinois, 60637

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • The Jewish Hospital

    Cincinnati, Ohio, 45236

  • Case Western Reserve University (Cwru) - University Hospitals of Cleveland (Uhc)

    Cleveland, Ohio, 44106

  • Robert J Tomsich Pathology & Laboratory Medicine Institute

    Cleveland, Ohio, 44195

  • Nationwide Children's Hospital - Ohio State University College of Medicine (OSUCOM)

    Columbus, Ohio, 43205

  • The Ohio State University, James Cancer Hospital

    Columbus, Ohio, 43210

  • Children's Medical Center of Dallas

    Dallas, Texas, 75235

  • Geisinger Medical Center

    Danville, Pennsylvania, 17822

  • US Oncology - Denver

    Denver, Colorado, 80218

  • Wayne State University/Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • Henry Ford Hospital

    Detroit, Michigan, 48202

  • City of Hope Medical Center

    Duarte, California, 91010

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • San Antonio Military Medical Center

    Fort Sam Houston, Texas, 78234

  • Cook Children's Health Care Hospital

    Fort Worth, Texas, 76104

  • UF Health Shands Hospital

    Gainesville, Florida, 32610

  • Helen DeVos Children's Hospital - Spectrum Health

    Grand Rapids, Michigan, 49503

  • Greenville Health System Cancer Institute

    Greenville, South Carolina, 29605

  • Hackensack University Medical Center - Joseph M. Sanzari Childrens Hospital

    Hackensack, New Jersey, 07601

  • Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Kapi'olani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Texas Children's Hospital

    Houston, Texas, 77030

  • The University of Texas - MD Anderson Cancer Center

    Houston, Texas, 77030

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202

  • Indiana Blood and Marrow Transplantation Clinical Lab LLC/St. Francis Hospital

    Indianapolis, Indiana, 46237

  • University of Iowa Hospitals & Clinics

    Iowa City, Iowa, 52242

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • University of Kansas Cancer Center

    Kansas City, Kansas, 66160

  • Children's Mercy Hospital and Clinics - Infectious Diseases

    Kansas City, Missouri, 64108

  • The University of California, San Diego

    La Jolla, California, 92037

  • University of Arkansas for Medical Services

    Little Rock, Arkansas, 72205

  • Loma Linda University

    Loma Linda, California, 92354

  • Long Beach Memorial Medical Center

    Long Beach, California, 90806

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027

  • Pediatric Blood and Cancer Disorders Clinic

    Louisville, Kentucky, 40202

  • University of Louisville Research Foundation

    Louisville, Kentucky, 40202

  • University of Wisconsin, American Family Children's Hospital, Medical School

    Madison, Wisconsin, 53792

  • Cardinal Bernardin Cancer-Loyola University Medical Center

    Maywood, Illinois, 60153

  • Methodist University Hospital

    Memphis, Tennessee, 38104

  • St. Jude Children's Research Hospital - Department of Bone Marrow Transplantation and Cellular Therapy

    Memphis, Tennessee, 38105

  • Jackson Memorial- University of Miami Health System- Pediatrics

    Miami, Florida, 33136

  • Nicklaus Children's Hospital (Miami Children's Hospital)

    Miami, Florida, 33155

  • Medical College of Wisconsin/ Children's Hospitalof Wisconsin

    Milwaukee, Wisconsin, 53226

  • Children's Hospitals and Clinics of Minnesota and Minneapolis

    Minneapolis, Minnesota, 55404

  • University of Minnesota Medical Center

    Minneapolis, Minnesota, 55455

  • Mary Babb Randolph Cancer Center - West Virginia University Hospital

    Morgantown, West Virginia, 26505

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203

  • Vanderbilt Ingram Cancer Center

    Nashville, Tennessee, 37232

  • Rutgers Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08903

  • Yale - New Haven Hospital

    New Haven, Connecticut, 06520

  • Steven and Alexandra Cohen Children's Medical Center

    New Hyde Park, New York, 11040

  • Tulane University Hospital & Clinic (Tuhc) - Tulane Cancer Center Comprehensive Clinic (Tcccc)

    New Orleans, Louisiana, 70112

  • Children's Hospital

    New Orleans, Louisiana, 70118

  • New York University School of Medicine

    New York, New York, 10016

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10021

  • Mount Sinai Hospital - BMT Program

    New York, New York, 10029

  • Mount Sinai Medical Center

    New York, New York, 10029

  • Morgan Stanley Children's Hospital - Columbia University Medical Center

    New York, New York, 10032

  • Weill Cornell Medical College - New York Presbyterian Hospital

    New York, New York, 10065

  • Children's Hospital of the King's Daughter

    Norfolk, Virginia, 23507

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609

  • The University of Oklahoma Health Science Center

    Oklahoma City, Oklahoma, 73104

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198

  • Children's Hospital of Orange County (CHOC)

    Orange, California, 92868

  • Florida Hospital

    Orlando, Florida, 42803

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • Mayo Clinic Cancer Center

    Phoenix, Arizona, 85054

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15219

  • West Penn Allegheny Health System

    Pittsburgh, Pennsylvania, 15224

  • Doernbecher Children's Hospital - Oregon Health and Science University

    Portland, Oregon, 97239

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of Rochester Medical Center, Strong Memorial Hospital

    Rochester, New York, 14642

  • Sutter Institute for Medical Research

    Sacramento, California, 95816

  • University of California Davis Health System

    Sacramento, California, 95817

  • University of Utah

    Salt Lake City, Utah, 84132

  • Methodist Healthcare System of San Antonio

    San Antonio, Texas, 78229

  • Rady's Children's Hospital - San Diego

    San Diego, California, 92123

  • University of California San Francisco Medical Center

    San Francisco, California, 94143

  • Arizona Oncology

    Scottsdale, Arizona, 85258

  • Fred Hutchinson Cancer Research Center/Seattle Children Hospital

    Seattle, Washington, 98109

  • Avera Cancer Institute

    Sioux Falls, South Dakota, 57105

  • Providence Sacred Heart Medical Center and Children's Hospital

    Spokane, Washington, 99204

  • Cardinal Glennon Children's Medical Center/St. Louis University Department of Pediatrics

    St Louis, Missouri, 63104

  • Washington University/St. Louis Children's Hospital

    St Louis, Missouri, 63110

  • All Children's Hospital Specialty Physicians

    St. Petersburg, Florida, 33701

  • Stanford University Medical Center

    Stanford, California, 94305

  • Stony Brook University Hospital

    Stony Brook, New York, 11794

  • H. Lee Moffitt Cancer Center & Research Institute

    Tampa, Florida, 33612

  • Montefiore Medical Center

    The Bronx, New York, 10467

  • University Medical Center

    Tucson, Arizona, 85724

  • New York Medical College

    Valhalla, New York, 10595

  • Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

  • Nemours-Al duPont Hospital for Children

    Wilmington, Delaware, 19803

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27157

Other trials in United States

About this study

Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.

Being tested
Defibrotide
Keywords
VOD
Medical subject headings
Hepatic Veno-Occlusive Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DefibrotideDrug

    Defibrotide is a single-stranded polydeoxyribonucleotide derived from porcine intestinal mucosa by controlled depolymerisation. Defibrotide has a complex mechanism of action with antithrombotic, anti-ischemic, anti-inflammatory, anti-adhesive and thrombolytic properties but no significant systemic anti-coagulant effects.

    Defibrotide is dose intravenously as a 2-hour infusion every 6 hours at a dose of 25 mg/kg/day. Recommended duration of therapy is 21 days.

  • DefibrotideDrug

Study groups in NCT00628498
Study groupRoleWhat they receive
DefibrotideExperimentalDefibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
Receives: Drug: Defibrotide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Survival by Day+100 Post Stem Cell Transplant or Chemotherapy

    Measured: Day +100 from HSCT or 100 days from start of chemotherapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Mar 2008 (estimated)
  2. Study startDec 2007
  3. Primary completionJul 2016
  4. Study completionSep 2016

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1,206 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00628498
Sponsor's protocol ID
P2006-05
Acronym
Treatment IND
Status last verified
Nov 2017
First submitted
25 Feb 2008
Last updated
30 Nov 2017
Data monitoring committee
No

Publications

  • Richardson PG, Murakami C, Jin Z, Warren D, Momtaz P, Hoppensteadt D, Elias AD, Antin JH, Soiffer R, Spitzer T, Avigan D, Bearman SI, Martin PL, Kurtzberg J, Vredenburgh J, Chen AR, Arai S, Vogelsang G, McDonald GB, Guinan EC. Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood. 2002 Dec 15;100(13):4337-43. doi: 10.1182/blood-2002-04-1216. Epub 2002 Aug 1.

    PubMed 12393437

  • Richardson PG, Smith AR, Kernan NA, Lehmann L, Ryan RJ, Grupp SA. Analysis of Time to Complete Response after Defibrotide Initiation in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation. Transplant Cell Ther. 2021 Jan;27(1):88.e1-88.e6. doi: 10.1016/j.bbmt.2020.09.008. Epub 2020 Sep 17.

    PubMed 32950693

  • Kernan NA, Grupp S, Smith AR, Arai S, Triplett B, Antin JH, Lehmann L, Shore T, Ho VT, Bunin N, Iacobelli M, Liang W, Hume R, Tappe W, Soiffer R, Richardson P. Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome. Br J Haematol. 2018 Jun;181(6):816-827. doi: 10.1111/bjh.15267. Epub 2018 May 16.

    PubMed 29767845

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Defibrotide in people with Hepatic Veno-Occlusive Disease, run by Jazz Pharmaceuticals at 125 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 125 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Nov 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Survival by Day+100 Post Stem Cell Transplant or Chemotherapy (time frame: Day +100 from HSCT or 100 days from start of chemotherapy). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00628498, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2017.

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